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Exon 1-targeting miRNA reduces the pathogenic exon 1 HTT protein in Huntington's disease models Sogorb-Gonzalez, Marina; Landles, Christian; Caron, Nicholas S.; Stam, Anouk; Osborne, Georgina; Hayden, Michael R.; Howland, David; van Deventer, Sander; Bates, Gillian P.; Valles, Astrid; Evers, Melvin 分享 收藏
Widespread and sustained target engagement in Huntington's disease minipigs upon intrastriatal microRNA-based gene therapy Valles, Astrid; Evers, Melvin M.; Stam, Anouk; Sogorb-Gonzalez, Marina; Brouwers, Cynthia; Vendrell-Tornero, Carlos; Acar-Broekmans, Seyda; Paerels, Lieke; Klima, Jiri; Bohuslavova, Bozena; Pintauro, Roberta; Fodale, Valentina; Bresciani, Alberto; Liscak, Roman; Urgosik, Dusan; Starek, Zdenek; Crha, Michal; Blits, Bas; Petry, Harald; Ellederova, Zdenka; Motlik, Jan; van Deventer, Sander; Konstantinova, Pavlina 分享 收藏
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Lipid nanoparticle pre-treatment improves adeno-associated virus diffusion in the primate liver and enables an increase of therapeutic transgene expression Kwikkers, Karin L.; Schwarz, Lukas; de Laat, Johannes; Majowicz, Anna; Miranda, Paula S.; Gielen, Alexander W.; de Haan, Martin; Petry, Harald; van Deventer, Sander; Ferreira, Valerie 分享 收藏
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SUSTAINED MUTANT HUNTINGTIN LOWERING IN THE BRAIN AND CEREBROSPINAL FLUID OF HUNTINGTON DISEASE MINIPIGS MEDIATED BY AAV5-MIHTT GENE THERAPY Valles, Astrid; Brouwers, Cynthia; Pintauro, Roberta; Snapper, Jolanda; Bohuslavova, Bozena; Sogorb-Gonzalez, Marina; Fodale, Valentina; Bresciani, Alberto; Ellederova, Zdenka; Blits, Bas; Motlik, Jan; van Deventer, Sander; Evers, Melvin; Konstantinova, Paylina 分享 收藏
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PRE-CLINICAL EVALUATION OF AAV5-MIHTT GENE THERAPY OF HUNTINGTON'S DISEASE IN RODENTS Miniarikova, Jana; Zanella, Ilaria; Blits, Bas; Evers, Melvin; Zimmer, Virginie; Spoerl, Alexia; Southwell, Amber; Hayden, Michael; van Deventer, Sander; Deglon, Nicole; Petry, Harald; Konstantinova, Pavlina 分享 收藏
Community crystal gazing (vol 34, pg 276, 2016) Acharya, Anu; Bingham, Kate; Bradner, Jay; Burke, Wylie; Charo, R. Alta; Cherry, Joel; Choulika, Andre; Coles, Tony; Cook-Deegan, Robert; Crook, Stanley T.; Diaz, Emilia; Erickson, Brent; Giddings, L. Val; Giwa, Sebastian Eriksson; Greenwood, James C.; Gulati, Vishal; Hall, Sam; Harris, John; Heywood, Jamie; Hill, Colin; Levin, Jeremy; Mangubat, Adina; Maraganore, John; Mariggi, Giovanni; Mazur, Barbara J.; McGuire, Amy L.; Moll, Nathalie; Moreno, Jonathan; Naughton, Gail; Nelsen, Lita; Osbourn, Jane; Perez, Daniel; Reed, John; Schmidt, Eric; Seyfert-Margolis, Vicki; Stoffels, Paul; Thorball, Jorgen; O'Toole, Tara; Vainu, Indrek; van Deventer, Sander; Zerhouni, Elias; Zohar, Daphne 分享 收藏
Community crystal gazing Acharya, Anu; Bingham, Kate; Bradner, Jay; Burke, Wylie; Charo, R. Alta; Cherry, Joel; Choulika, Andre; Coles, Tony; Cook-Deegan, Robert; Crook, Stanley T.; Diaz, Emilia; Erickson, Brent; Giddings, L. Val; Giwa, Sebastian Eriksson; Greenwood, James C.; Gulati, Vishal; Hall, Sam; Harris, John; Heywood, Jamie; Hill, Colin; Levin, Jeremy; Mangubat, Adina; Maraganore, John; Mariggi, Giovanni; Mazur, Barbara J.; McGuire, Amy L.; Moll, Nathalie; Moreno, Jonathan; Naughton, Gail; Nelsen, Lita; Osbourn, Jane; Perez, Daniel; Reed, John; Schmidt, Eric; Seyfert-Margolis, Vicki; Stoffels, Paul; Thorball, Jorgen; O'Toole, Tara; Vainu, Indrek; van Deventer, Sander; Zerhouni, Elias; Zohar, Daphne 分享 收藏
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Tolerability, pharmacokinetics and pharmacodynamics of TA-8995, a selective cholesteryl ester transfer protein (CETP) inhibitor, in healthy subjects Ford, John; Lawson, Matt; Fowler, David; Maruyama, Nobuko; Mito, Seiji; Tomiyasu, Koichi; Kinoshita, Shuji; Suzuki, Chisa; Kawaguchi, Atsuhiro; Round, Patrick; Boyce, Malcolm; Warrington, Steve; Weber, Werner; van Deventer, Sander; Kastelein, John J. P. 分享 收藏
Embedding siRNA sequences targeting Apolipoprotein B100 in shRNA and miRNA scaffolds results in differential processing and in vivo efficacy Maczuga, Piotr; Lubelski, Jacek; van Logtenstein, Richard; Borel, Florie; Blits, Bas; Fakkert, Erwin; Costessi, Adalberto; Butler, Derek; van Deventer, Sander; Petry, Harald; Koornneef, Annemart; Konstantinova, Pavlina 分享 收藏
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Molecular Therapy for Obesity and Diabetes Based on a Long-Term Increase in Hepatic Fatty-Acid Oxidation Orellana-Gavalda, Josep M.; Herrero, Laura; Ida Malandrino, Maria; Paneda, Astrid; Sol Rodriguez-Pena, Maria; Petry, Harald; Asins, Guillermina; Van Deventer, Sander; Hegardt, Fausto G.; Serra, Dolors 分享 收藏
In vitro and in vivo down-regulation of regulatory T cell activity with a peptide inhibitor of TGF-β1 Gil-Guerrero, Lucia; Dotor, Javier; Huibregtse, Inge Louise; Casares, Noelia; Belen Lopez-Vazquez, Ana; Rudilla, Francesc; Ignacio Riezu-Boj, Jose; Lopez-Sagaseta, Jacinto; Hermida, Jose; Van Deventer, Sander; Bezunartea, Jaione; Llopiz, Diana; Sarobe, Pablo; Prieto, Jesus; Borras-Cuesta, Francisco; Jose Lasarte, Juan 分享 收藏
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